safina adatia: rare disease day 2016 conference
TRANSCRIPT
CIHRIns(tuteofGene(cs
SafinaAda(a,MScProjectOfficer|Agentedeprojets
CIHRIns(tuteofGene(cs|Ins(tutdegéné(quedesIRSCsafina.ada([email protected]
@SAda(a
StrategicPlan(2012-17)
• StrengtheningtheIGResearchCommunity
• AddressingHealth&HealthSystemPriori(es:– Computa(onalandSystemsBiology– FromModelsandMechanismstoTherapies– HealthServices,Policy&Ethical,LegalandSocialIssuesResearch
• FosteringInnova(on,Commercializa(on&Transla(onThroughPartnerships
CIHRPersonalizedMedicineSignatureIni7a7ve
Enhancehealthoutcomesthroughpa(entstra(fica(onapproachesbyintegra(ngevidence-basedmedicine&
precisiondiagnos(csintoclinicalprac(ce
develop&translatediscoveries
biomarkers,targets&genomicsignatures
diagnos(cs&innova(vedevicesforclinicaluse
supportpolicy&prac(ce
improvetheevidencebaseonhowtoassess&eventually
integrateinnova(vediagnos(cs&therapeu(capproachesintoprac(ce
CanadianPersonalizedMedicineStakeholders
Federal
Pa(ent/ProfessionalGroups
Provinces&Territories Industry
Research
CIHR/NSERC/GenomeCanada
ResearchPriori(es
Na(onalLabStandards
ProductR&D
PMPRBProduct
Submissions
Surveillance
CADTH
HealthCanada
PHAC
Na(onalPolicyInterests
Provincial&TerritorialResearch
FormularyDiscussions
Provincial&TerritorialLabStandards
T.RyanSigouin,HealthCanadaadaptedbyIngaMurawskiCIHR-ICRandE(enneRicherCIHR-IG
HealthcarePriori(es
INESSS
PartnershipsSummary
Budget–FirmandPlannedInvestments
CIHR/Genome Canada partnership: One of the most significant public sector investment in PM
• Research projects span various areas including cancer, rare diseases, epilepsy, inflammation, HIV, cardiovascular disease and autism
Investment: over $165M • $68.8M CIHR/GC investment with more 1:1 match from outside
sources Projects funded: 17 • 15 large-scale applied research projects with integrated GE3LS • 2 large-scale GE3LS research projects
GenomicsandPersonalizedHealth:2012Large-ScaleAppliedResearchProjectCompe((on
CIHR’s contribution was made available by the following Institutes (IG, ICR, ICRH, INMHA, III, INMD & IHSPR) & Initiatives (HIV/AIDS & Breast Cancer)
E-Rare-3:ERA-NETCo-FundApplica7on
JointTransna7onalCall(JTC)2015:• inpartnershipwiththreeCdnVHOs:MDC,ARSACS&CFC• fundsavailableforCdncomponents:upto$3.75M• objec(ve:toenablescien(stsindifferentcountriestobuild
aneffec(veresearchcollabora(on,involvingagroupofrarediseasesorasingleraredisease
Compe77ondetails:• Applica(ons:51(14withaCanadianco-applicant)• CurrentlySelectedforFunding:21(9withaCanadianco-
applicant)
26researchfunders(from18countries)coordina(ngrarediseaseresearchfundingthroughtransna(onalcallsdesignedtoimplementIRDiRCobjec(ves&priori(es
E-Rare3JTC2016• Currentcalltopic:
– Clinicalproofofconceptforthepoten(alapplica(oninrareindica(onsofmedicinalproductseitheralreadymarketedorhavingachievedasignificantstageinthedevelopmentprocess.
• An(cipatedfundingstartdate:Early2017• Budget:$2,5Mover3years(CIHR-IG)• Effortsunderwaytoincludethepar(cipa(onofotherCanadianfundingpartners
NorthAmericanRe:Rare• RepurposingResearchinRareDiseases:CuresWithin
Reach/CIHRCollabora(veFundingProgram• Goal:tosupporteffortsonproofofconceptclinicaltrials
repurposinghumanapproveddrugsfornewdiseaseindica(ons
• Proposalsrequirements:– generatecleardeliverableswithimmediateapplica(ons,
includingoff-labeluse,forrarediseasesmanagement;– proposenovel(evalua(oncriteriatolowerprojectsthathave
beendone)proofofconceptclinicaltrialsinvolving,mul(pleclinicalsitesinCanadaandtheUnitedStatesofAmerica,and;
– proposeanadequatedevelopmentplantoensuretherealiza(onoftheexpectedbenefits.
• Budget:$1.5MUSD(CIHRandCWR)/3years
Interna7onalRareDiseasesResearchConsor7um
• Foster international collaboration in rare diseases research ü Deliver 200 new therapies for rare diseases and
Ø 155 as of September 18th
ü Means to diagnose most rare diseases by the year 2020 Ø About 3500 as of September 18th
• 43 funding members (including E-Rare) & 3 invited patient advocacy groups
• Minimal commitment: US$10 million over five years in research projects contributing towards the goals of IRDiRC
• CIHR & Genome Canada joint commitment of $25 million to IRDiRC while the
• Total pledges of consortium nearly $2 billion
10
OrphanetCanada
• Toprovidethecommunityatlargewithacomprehensivesetofinforma(oninordertocontributetotheimprovementofthediagnosis,careandtreatmentofpa(entswithrarediseases.
Mission
• Createana(onalentrysitetocommunicateatthena(onallevelonac(vi(esofthena(onalOrphanetteam,rarediseaseevents,andrarediseasepoliciesinthecountry
• ComprehensivelydocumentresourcesavailableinCanada:expertcenters,medicallaboratories,pa(entorganisa(on,researchprojects,clinicaltrials,andregistriesandbiobanks
• OutreachtoallstakeholdersoftheOrphanet-Canadaprojectandfacilitateexchangesbetweengroupofstakeholders
Na(onalobjec(vesareto
Resource Completed Expected
Clinics 47 -
Diagnostic tests
487 25
Patient Organizations
78 135
Clinical Trials 27 34+
Registries 8 16
Research Projects
148 155
